Ascendis Pharma is a biopharmaceutical company developing its TransCon technology to create long-acting prodrug therapies. It has three drug candidates in clinical trials for growth hormone deficiency, hypoparathyroidism, and achondroplasia. Its lead candidate, TransCon hGH, is in Phase 3 trials for growth hormone deficiency and top-line data is expected in Q1 2019. Analysts have rated Ascendis Pharma stock a "Buy" based on the potential of its TransCon technology to improve treatment outcomes for multiple rare diseases.

1. Zachary Hensley
&
Connor Roberts

2. Primary Stock
Information
Exchange: NASDAQ-GS
Ticker: ASND
Sector: Health Care
Industry: Biotechnology/Biopharmaceuticals
Market Cap: $3.033B
Current Stock Price: $71.99 (Feb 22, 2019 @
4:00 PM)
52 Week Range: $53.21-$76.99
Shares Outstanding: 42.14M
PE: NM
Forward PE: NM
PEG: NM
Beta(3Y): 1.32

3. Price Movement: Ascendis Pharma A/S v. S&P Biotechnology v. S&P 500 (1y)

4. Price Movement: Ascendis Pharma A/S v. Competitors v. S&P Biotechnology (1y)

5. Price Movement: Ascendis Pharma A/S v. Competitors v. S&P Biotechnology (3y)

6. Industry Outlook: Biotechnology
⃟ 2019 year-over-year growth rate is estimated at 5.98%
⃟ Risk of Drug Price Regulation
⬩ move towards legislating direct drug price negotiating power for the Centers for Medicare & Medicaid
Services as a tactic to reduce drug prices.
⃟ Biotech Pipeline Remains Rich With Potential
⬩ This has combined with a more active Federal Drug Administration (FDA) focused on shrinking decision-
making timelines, to create numerous success stories during 2018.
⬩ As of mid-December 2018, the FDA had approved 56 new novel drugs
⃟ Mergers & Acquisitions - Lower Valuations May Spark Activity
⬩ Low prices mean M&A opportunities
⬩ The M&A drought in 2018 was during a period of very easy financing options for biotechs and record
access to public markets through IPOs. There have been 58 biotech IPOs in 2018 raising over $6 billion
⬩ Ensuing bear market in biotechs with moderating valuations will rekindle interest during 2019 in the
pipeline assets of biotechs
⬩ Smaller companies that hold important patents could be acquired by the larger companies to gain an
“edge in this competitive market

7. Industry Outlook: Biotechnology

8. Trial Phases & Process to Approval
Phase 1: The drug is initially introduced into healthy human subjects or patients with the target disease or condition
and tested for safety, dosage tolerance, absorption, metabolism, distribution, excretion and, if possible, to gain an
early indication of its effectiveness.
Phase 2: The drug is administered to a limited patient population to identify possible adverse effects and safety risks,
to preliminarily evaluate the efficacy of the product for specific targeted diseases and to determine dosage tolerance
and optimal dosage.
Phase 3: The drug is administered to an expanded patient population, generally at geographically dispersed clinical
trial sites, in well- controlled clinical trials to generate enough data to statistically evaluate the efficacy and safety of
the product for approval, to establish the overall risk- benefit profile of the product, and to provide adequate
information for the labeling of the product.
Phase 4: In some cases, the FDA may condition approval of an NDA for a product candidate on the sponsor's
agreement to conduct additional clinical trials after NDA approval. In other cases, a sponsor may voluntarily conduct
additional clinical trials post approval to gain more information about the drug. Such post approval trials are typically
referred to as Phase 4 clinical trials.

9. Growth Hormone Deficiency (GHD)
⃟ GHD results from inadequate secretion of growth hormone (GH) from the anterior pituitary
gland (the gland responsible for hormone secretion).
⃟ GHD is characterized by short stature, metabolic abnormalities, & cognitive, cardiovascular,
muscular, and skeletal abnormalities.
⃟ Mostly diagnosed in children between the ages of 5 & 16 (pre-puberty).
⃟ Affects ~ 1 out of every 7,000 born
Current Treatments: Once-daily injections from the following drugs:
⃟ Genotropin (Pfizer)
⃟ Norditropin (Novo Nordisk)
⃟ Humatrope (Eli Lilly & Co.)

10. Hypoparathyroidism (HP)
⃟ HP stems from inadequate parathyroid
hormone production.
⃟ Those with HP have decreased calcium levels
and abnormally high phosphate levels in their
blood (this may cause kidney disease).
⃟ Can cause cognitive, physical, and emotional
symptoms (depression, psychiatric disorders).
Patients may also experience seizures (and
death, as a result).
⃟ 75% of cases are a direct result of damage to
the parathyroid during neck surgery.
⃟ ~ 80,000 U.S. Patients

11. Achondroplasia (Dwarfism)
⃟ Caused by imbalance of C-type natriuretic peptide (CNP) and mutations in the FGFR3 gene --
resulting in growth plate suppression.
⃟ Affects ~250,000 patients worldwide
⃟ 45,000 new cases reported annually (extremely prevalent in Denmark).
⃟ Currently, there are no medical therapies approved/available for those suffering from
Achondroplasia.

12. Growth Hormone Market

13. Business Description

14. Recent News
⃟ February 27, 2019 - EPS for Ascendis Pharma A/S (ASND) Expected At
$(1.18)
⃟ February 11, 2019 - Ascendis Pharma Initiates Global Phase 2 Trial for
TransCon PTH in Adult Subjects with Hypoparathyroidism
⃟ February 9, 2019 - Ascendis Pharma inks $200M stock sales deal with
JPMorgan
⃟ January 28, 2015 – Ascendis Pharma raised $85 million in an IPO.

15. Competitors

16. Novo Nordisk A/S
⃟ Novo Nordisk manufactures and markets pharmaceutical products and
services.
⃟ Key products include diabetes care medications and devices. Novo
Nordisk is also involved with hemostasis management, growth hormone
therapy and hormone replacement therapy.
⃟ The company makes several drugs under various brand names, including
Levemir, Tresiba, NovoLog, Novolin R, NovoSeven, NovoEight and Victoza.

17. Pfizer, Inc.
⃟ Develops and produces medicines and vaccines for a wide range of medical disciplines,
including immunology, oncology, cardiology, endocrinology, and neurology.
⃟ American pharmaceutical corporation headquartered in New York City.
⃟ Research headquarters in Groton, Connecticut.
⃟ One of the world's largest pharmaceutical companies.
⃟ Products include: Lipitor (atorvastatin), used to lower LDL blood cholesterol; Lyrica
(pregabalin) for neuropathic pain and fibromyalgia; Diflucan (fluconazole), an oral antifungal
medication; Zithromax (azithromycin), an antibiotic; Viagra (sildenafil) for erectile dysfunction;
and Celebrex (also Celebra, celecoxib), an anti-inflammatory drug.

18. Equity Ownership

19. Corporate Governance
Top Executives
Mr. Michael Wolff Jensen – Chairman, Sr. VP & Chief Legal Officer
Mr. Jan Møller Mikkelsen – Pres, CEO, Member of Exec. Board & Exec.
Director
Mr. Scott T. Smith - CFO, Sr. VP & Member of Exec. Board
Mr. Peter Rasmussen - VP of Fin. & Principal Accounting Officer
Ms. Lotte Sønderbjerg - Chief Admin. Officer & Sr. VP
Dr. Harald Rau – Sr. VP, Chief Scientific Officer

20. Mr. Jan Møller Mikkelsen
⃟ CEO, President, Executive Director and Member of Executive Board of Ascendis Pharma A/S.
⬩ Chairman of HyScite Discovery & Director of Ascendis Pharma A/S
⃟ 1989-1999, served at Novo Nordisk A/S
⬩ Director of Protein Discovery, heading a multinational organization of more than 150 people and was a
member of enzyme research management.
⃟ Former President of Maxygen's Protein Pharma Division and Managing Director of Maxygen APS
⃟ Former CEO and President at Veloxis Pharmaceuticals A/S until August 2006.
⃟ Former Member of Advisory Team at Nordic Biotech.
⃟ Served as a Director of Nuevolution A/S

21. Dr. Harald Rau
⃟ Sr. VP, Chief Scientific Officer, managing the research group at Ascendis Pharma since
December 2007.
⃟ Served as the Chief Scientific Officer of Complex Biosystems GmbH, a biotechnology company
he co-founded in 2002.
⬩ Acquired by Ascendis Pharma in 2007
⃟ Worked at Graffinity Pharmaceuticals AG (1998-2002)
⬩ Biotechnology company
⬩ Served as Director of Chemistry from 2000-2002.

22. Stock Performance Risk Factors
1. Delayed Cash Flows from Delayed Product Development/Approval
2. Scalable Manufacturing & Supply Chain (Availability of Supply to Meet Demand)
3. Collaboration Partners’ Ability to Commercialize & Distribute Product (Marketing)
4. Efficacy + Side Effects Related to Products
5. Third-Party Payers (Coverage + Reimbursement Policies)/Health Care Reform
(U.S.)
6. Running out of Cash (Highly-unlikely to take place)
7. Diluting Current Shares; Raising Capital by Issuing More Shares
8. Litigation/Settlements
9. Global Political Risks (Brexit, Emerging Markets)
10. Intellectual Property Theft
11. Import + Export Regulations
12. Identifying, Retaining, Incentivizing, and Integrating New Employees
13. Exchange Rate Risk (Euro, Danish Krone, USD)

23. Costs Associated with Operations
⃟ Research & Development
⃟ Regulation (Compliance), Lobbying
⃟ Marketing
⃟ Distribution
⃟ Outsourcing (Manufacturing + Strategic Partnerships)
⃟ Litigation
⃟ Highly-Skilled Personnel

24. Cash On-Hand

25. Research & Development

26. What is TransCon Technology?

27. Products in Development (All of Them)
⃟ TransCon hGH
⬩ Once-weekly therapy injection treatment for GHD
⬩ Currently in Phase III of Clinical Trials
⬩ Expect top-line data from Q1 2019
⃟ TransCON PTH
⬩ Once-daily, long acting injectable prodrug treatment for HP
⬩ Recently, was approved for Phase II of Clinical Trials (unveil top-line data Q4 2019)
⃟ TransCon CNP
⬩ Once-daily treatment for achondroplasia
⬩ Delivers C-type natriuretic peptide (CNP) to help balance FGFR3
⬩ Commenced Phase I of Clinical Trials in Dec. 2017 (Data Supported Efficacy)
⬩ Phase II initiation in Q3 2019

28. Clinical Trial Data (TransCon hGH)

29. Clinical Trial Data (TransCon hGH)

30. Clinical Trial Data (TransCon PTH)

31. Clinical Trial Data (TransCon PTH)

32. Clinical Trial Data (TransCon CNP)

33. Clinical Trial Data (TransCon CNP)

34. Suppliers

35. Vision 3x3: Strategic Roadmap to 2025

36. Manufacturing, Marketing, & Distribution
4321

37. Competitive Advantages/Market Opportunities
Associated with TransCon Technology
Enhance:
1. Efficacy
a. Enables Systemic Drug Exposure
b. Reduces Dosing Frequency
c. Optimizes Parent Drug Effects
2. Safety & Tolerability
a. Establishes Safety of Parent Drug
b. Allows Drug Enhancement/Innovation Potential
3. Development & Regulatory
a. Higher Development Success Rate of Parent Drug
b. Expedites Development Pathways (Through Existing Knowledge)

38. Competitive Advantages/Market Opportunities
Associated with TransCon Technology

39. Public Offerings/Shelf Registration History

40. Ascendis Pharma Current Revenue

41. Future Revenue Estimates

42. EBIT Estimates

43. Net Income Estimates

44. Earnings Per Share Estimates

45. Free Cash Flow

46. EBIT & Profit Margins

47. Analyst Estimates
Analyst
Consensus:
BUY

48. Intrinsic Value

49. BUY!!!

50. Sources
⃟ https://rarediseases.org/rare-diseases/growth-hormone-deficiency/
⃟ https://www.healthline.com/health/growth-hormone-deficiency
⃟ https://www.endocrineweb.com/conditions/hypoparathyroidism/hypoparathyroidism
⃟ https://rarediseases.info.nih.gov/diseases/8173/achondroplasia
⃟ https://ascendispharma.com/platform/transcon-technology/
⃟ https://money.cnn.com/quote/forecast/forecast.html?symb=ASND
⃟ https://ascendispharma.gcs-web.com/static-files/c3a5a97b-b615-485f-b7b9-243edb85aae2
⃟ https://www.capitaliq.com/CIQDotNet/Filings/DocumentRedirector.axd?versionId=291435172&type=pdf&forcedownload=false